University of Oklahoma Health Sciences Center

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Author/s:

Kareem Jamani, Ashley Chopek, Rajat Kumar, Gregory M.T. Guilcher

Date Added:

April 2, 2026

Journal/Publication:

Canadian Medical Association Journal

Publication Date:

March 29, 2026

URL:

Transformative therapies for sickle cell disease

Type:

Meta-analyses, Reviews, and Guidelines

Format:

Article

DOI (1):

10.1503/cmaj.251338

PMID (1):

41912244

Keywords

Sickle cell disease

Sickle cell

Sickle cell therapies

allogeneic hematopoietic stem cell transplant

gene therapy

Hematopoietic Stem Cell Transplantation

MeSH

Sickle Cell

allogeneic hematopoietic stem cell transplant

gene therapy

Hematopoietic Stem Cell Transplantation

RPR Commentary

A concise review of stem cell and gene therapy options for patients with sickle cell disease. James W. Mold, MD, MPH

Abstract

Sickle cell disease (SCD) is associated with major morbidity, reduced life expectancy, and impaired quality of life.

Transformative therapies for SCD include allogeneic hematopoietic stem cell transplant and gene therapy.

After successful transformative therapy, people with SCD no longer demonstrate an SCD phenotype and experience a reduction in SCD-related morbidities and improvement in quality of life.

Transformative therapies for SCD can be associated with major adverse events, including death.

The decision to proceed with a transformative therapy must take into careful consideration the severity of the SCD, the risk of future SCD complications, and the risks of available transformative therapies.

The expertise and infrastructure to deliver transformative therapies lies within existing Canadian cancer care programs.